Effect of sorafenib on prognosis in patients with low-allelic-ratio FLT3-ITD acute myeloid leukemia undergoing allogeneic hematopoietic stem cell transplantation

Abstract

Background: The effect of sorafenib in acute myeloid leukemia (AML) patients with low-allelic-ratio FMS-like tyrosine kinase 3 internal tandem duplication(FLT3-ITD^{low AR}) is still unclear.

Objective: The purpose of this study was to evaluate whether sorafenib can improve prognosis in FLT3-ITD^{low AR} AML patients.

Study design: In this study, the effect of sorafenib on the prognosis of patients with FLT3-ITD^{low AR} AML was retrospectively explored. A total of 112 patients with FLT3-ITD^{low AR} AML who underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT) were enrolled, including 44 patients who received sorafenib treatment and 68 patients who did not receive sorafenib.

Results: With a 27-month median follow-up, there was no significant difference in overall survival (OS) or Relapse-free survival (RFS) between the sorafenib and control groups (2-year OS: 54.4 % vs. 52.3 %, p = 0.64; 2-year RFS: 49.2 % vs. 36.6 %, p = 0.25). However, for patients with refractory/relapsed (R/R) disease (sorafenib group: n = 29, control group: n = 20), patients in the sorafenib group had better 2-year OS (2-year OS: 22.7 % vs. 15 %, p = 0.018) and RFS (2-year RFS: 36.4 % vs. 10 %, p = 0.051) than did those in the control group. Additionally, multivariate analysis confirmed that receiving sorafenib therapy was an independent protective factor for OS in R/R FLT3-ITD^{low AR} AML patients.

Conclusion: Overall, the use of sorafenib could improve outcomes in R/R AML patients with FLT3-ITD^{low AR} mutations.

Keywords: Acute myeloid leukemia; Allogeneic hematopoietic stem cell transplantation; FLT3-ITD(low AR); Refractory/relapsed; Sorafenib.